What is the role of clofarabine in the treatment of acute lymphoblastic leukemia (ALL)?

Updated: Oct 26, 2020
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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In December 2004, the US Food and Drug Administration (FDA) granted accelerated approval for clofarabine, a novel nucleoside analogue, for the treatment of pediatric patients with refractory or relapsed ALL. Two open-label, multicenter, nonrandomized phase II trials established the efficacy and safety profile of clofarabine in that patient population. In one study of 61 patients (median age, 12 years; range, 1-20 years), the response rate to clofarabine was 30% (seven CRs, five CRs without platelet recovery, and six partial remissions), and remissions lasted long enough to allow patients to proceed to hematopoietic stem-cell transplantation (HSCT). [72]

In a second study in 42 patients (median age, 13 years; range, 2-22 years), the response rate was 26% and included one complete response without platelet recovery and 10 partial responses. The median duration of response was 20 weeks. [73]

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